orBec® - for the treatment of gastrointestinal symptoms associated with chronic Graft-versus-Host disease

orBec® is specifically formulated for oral administration as a single product consisting of two tablets (each containing 1 mg of beclomethasone 17,21-dipropionate or BDP). One tablet releases BDP in the upper gastrointestinal (GI) tract and the other tablet releases BDP in the lower GI tract. BDP is a highly potent, topically active corticosteroid. The Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to BDP for the treatment of GI symptoms associated with chronic Graft-versus-Host disease (cGVHD) in patients who have undergone allogeneic hematopoietic cell transplantation.

About Chronic GVHD

Graft-versus-Host disease (GVHD) is a major complication of allogeneic hematopoietic cell transplantation. GVHD is an inflammatory disease initiated by T cells in the donor graft that recognize histocompatibility and other tissue antigens of the host, and is mediated by a variety of effector cells and inflammatory cytokines. GVHD presents in both acute and chronic forms. The symptoms of cGVHD typically present at between 100 days and three years post-transplant.

Chronic GVHD has features resembling autoimmune and other immunologic disorders such as scleroderma, Sjögren syndrome, primary biliary cirrhosis, wasting syndrome, bronchiolitis obliterans (BO), immune cytopenias and chronic immunodeficiency. The manifestations of cGVHD may be restricted to a single organ or tissue or may be widespread. Chronic GVHD can lead to debilitating consequences, e.g., joint contractures, loss of sight, end-stage lung disease, or mortality resulting from profound chronic immune suppression leading to recurrent or life-threatening infections.

Treatment of chronic GI GVHD is a challenge because it can be refractory to frontline immunosuppression. High-dose systemic corticosteroids are used with some success but carry significant toxicity. The risks of prolonged immunosuppression include local and disseminated infections, Epstein-Barr virus associated lymphoproliferative disease, hypothalamic-pituitary-adrenal (HPA) axis suppression, myopathy, glucose intolerance, neuropsychiatric disease and bone demineralization.

Chronic GI GVHD affects approximately 6,000 patients annually in the United States and an approximately equal amount in the rest-of-world.